Nevertheless, classical transgenic mice were instrumental to show that PML-RARA indeed is the genetic driver of APL and to study the underlying molecular mechanisms leading to the first (and so far only) really efficient targeted AML therapy based PML-RARA degradation by all-trans-retinoic acid (ATRA) and/or arsenic trioxide [54]. The gene discussed is PML; the disease is acute myeloid leukemia.